Clinical and biomarker characteristics of Huntington’s Disease patients recently proposed for huntingtin-lowering clinical trial inclusion
Objective: We investigated plasma NfL levels, as well as clinical measures, in Huntingtin (Htt)-mutation carriers 48 years of age and under, with a CAP score…Neuroprotective trial design methodology for Parkinson’s: a scoping review
Objective: To further understanding of the diversity and impact of trial design choices in trials of disease modifying therapy (DMT) in Parkinson’s disease (PD). Background:…Vodobatinib, a potent, orally bioavailable brain-penetrating inhibitor of c-Abl as a potential neuroprotective agent for the treatment of Parkinson’s Disease
Objective: To compare and contrast nilotinib and vodobatinib regarding efficacy, brain penetration, and safety. Background: Preclinical evidence suggests that c-Abl is critical for pathogenesis of…An AAV9 encoding human ABCD1 (SBT101) shows functional improvement following spinal cord delivery in a rodent model of adrenomyeloneuropathy
Objective: SBT101 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy candidate delivering a functional copy of the human ABCD1 gene that is in development…Nutritional Status of Patients with Parkinson’s Disease: Report from Indonesian Tertiary Referral Hospital
Objective: This study aimed to describe and determine the prevalence of nutritional status in outpatient Parkinson’s Disease (PD) patients. Background: Parkinson’s disease is the most…Clinical Manifestations of Metabolic Syndrome in Parkinson’s Disease – Report from Indonesia Tertiary Referral Hospital
Objective: The study aims to determine the prevalence of metabolic syndrome and related factors in Parkinson's disease (PD). Background: Parkinson's disease is a neurodegenerative disorder,…Sex differences in vascular risk factors for Parkinson’s disease
Objective: Our aim is to illustrate the incidence of vascular risk factors (VRF) in a cohort of patients with confirmed Parkinson's disease (PD) and characterize…GT-02287, a brain-penetrant structurally targeted allosteric regulator for glucocerebrosidase show evidence of pharmacological efficacy in conduritol β-epoxide (CBE) models of Parkinson’s disease
Objective: Demonstrate the involvement of our structurally targeted allosteric regulator Parkinson’s disease (PD) candidate, GT-02287, in decreasing the CBE-induced neurotoxic effect and in restoring lysosomal…Designing a multi-arm multi-stage trial for Parkinson’s
Objective: The Edmond J Safra ACT-PD initiative (EJS ACT-PD) is developing the first multi-arm multi-stage (MAMS) platform trial for potential disease modifying approaches for Parkinson’s…Fatty acids and Glucocerebrosidase activity as potential biomarkers of stearyl-CoA desaturase inhibition; an observational study of inter and intraday variation in healthy volunteers and patients with Parkinson’s disease
Objective: This study aimed to assess the naturally occurring levels and variation in plasma fatty acids (FA) and ceramide metabolism in healthy volunteers (HV) and…
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